Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...
The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
How CRISPR therapy could cure everything from cancer to infertility The imminent approval of the world's first CRISPR treatment for sickle cell disease is just the start: soon this gene-editing ...
Gene therapy has been available since 2022 for ... “We would call that the low-to-mild range. By definition, there are people that are higher and lower.” Patients must have serious disease ...
The Metachromatic Leukodystrophy Initiative (MLDi) critically responds to the findings of Zhang et al. on lentivirus-modified ...
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New grant funds first-of-its-kind gene therapy to treat aggressive brain cancerThe California Institute for Regenerative Medicine has awarded a $6 million grant to USC investigators pioneering a new first-of-its-kind genetic therapy for glioblastoma, a severe form of brain ...
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